AveXis Releases Favorable Data in Ongoing Phase 1 Test of SMA Gene Therapy, AVXS-101
AveXis recently reported favorable results from an ongoing Phase 1 clinical trial examining its candidate, AVXS-101, for the systematic delivery of…
Articles by Ana de Barros, PhD
Regular Exercise Seen to Protect Motor Neurons in Spinal Muscular Atrophy Animal Study
Long-term exercise was found to benefit mice models of spinal muscular atrophy (SMA), providing important clues for designing rehabilitation programs in…
Muscle Activators That Might Treat SMA at Forefront of Company’s 5-Year Plan
Cytokinetics Inc. has presented its Vision 2020: Empowering Our Future — an initiative designed to expand the company’s drug pipeline and…
In Spinal Muscular Atrophy, Vascular System May Play a Key Role
For the first time, scientists have shown that insufficient blood supply is likely to contribute to motor neuron loss in spinal…
Cure SMA Publishes Clinical and Regulatory Webinar Q&A
The non-profit organization Cure SMA has published the video and transcription of an event recently organized, titled “Clinical Regulatory…
Cure SMA’s Medical Advisory Council Sets New Care Agenda
Members of Cure SMA’s Medical Advisory Council (MAC) recently met for two days in Chicago to set the agenda for the care of…
Cure SMA Given 10 New Feeder Seats By Jadon’s Hope Foundation
Cure SMA has been given 10 special tomato feeder seats by the Jadon’s Hope Foundation. The donation is meant to help…
Initial Results of Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress
AveXis, Inc. has recently presented results from its ongoing Phase 1/2 trial of AVXS-101 for the treatment of spinal muscular…
Genentech Updates Clinical Development of RG7800 for SMA
Genentech, a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious or life-threatening…
Low-Dose Irradiation to Edit The Genome of Patients’ Stem Cells
In a recent study published in the journal Stem Cells Translational Medicine, a team of researchers from…